18618604. RNA EDITING simplified abstract (HOFFMANN-LA ROCHE INC.)
Contents
RNA EDITING
Organization Name
Inventor(s)
Lars Joenson of Viby Sjaelland (DK)
Tobias Merkle of Copenhagen (DK)
Anja Moelhart Hoeg of Hillerød (DK)
Jonas Vikesaa of Fredensborg (DK)
RNA EDITING - A simplified explanation of the abstract
This abstract first appeared for US patent application 18618604 titled 'RNA EDITING
The present invention involves an antisense oligonucleotide designed to treat and/or prevent polyglutamine (polyQ) diseases by targeting a specific region of mRNA encoding a disease-related protein.
- Specifically binds to a CAG repeat region of the mRNA.
- Forms a double-stranded RNA that attracts Adenosine Deaminase Acting on RNA (ADAR) to insert an A to I exchange into the CAG repeat region.
- Pharmaceutical composition includes the antisense molecule.
Potential Applications: - Treatment of polyQ diseases such as Huntington's disease. - Prevention of disease progression in individuals at risk for polyQ diseases.
Problems Solved: - Targeted approach to modifying disease-causing mRNA. - Potential to slow down or halt disease progression.
Benefits: - Precision medicine approach for polyQ diseases. - Potential to improve quality of life for affected individuals.
Commercial Applications: - Development of targeted therapies for polyQ diseases. - Pharmaceutical companies can leverage this technology for drug development.
Questions about Antisense Oligonucleotide for PolyQ Diseases: 1. How does the antisense oligonucleotide specifically target the CAG repeat region of the mRNA? 2. What are the potential challenges in translating this technology into clinical applications?
Frequently Updated Research: - Stay updated on clinical trials testing the efficacy of antisense oligonucleotides in treating polyQ diseases.
Original Abstract Submitted
The present invention relates to an antisense oligonucleotide for use in treating and/or preventing a polyglutamine (polyQ) disease, wherein said antisense oligonucleotide is capable of specifically binding to a CAG repeat region of an mRNA encoding a polyQ disease-related protein such that a double stranded RNA is formed which is capable of attracting an Adenosine Deaminase Acting on RNA (ADAR) inserting an A to I exchange into at least one CAG trinucleotide of the CAG repeat region of said mRNA. The present invention further relates to a pharmaceutical composition comprising said antisense molecule.
